Check for an Expanded Access Program (EAP)

If you learn that new drug you want is available in an Expanded Access Program (EAP) you must find out if you qualify to enter the EAP. Most drug companies sponsoring Expanded Access Programs also sponsor an 800-telephone service to assist patients in determining their eligibility for the EAP.

The 800 numbers can often be found on the drug company's web site or by calling FDA's Office of Special Health Issues - Cancer Liaison Program at 301-827-4460. If the patient meets the EAP entry criteria, the drug company will provide the information about the sites where the patient may get the drug.

Ask about Single Patient Access

If an Expanded Access Program doesn't exist or if you don't qualify for it, then you can ask the company to supply the drug on a Single Patient Access basis. If the drug company says they are restricting use of their new drug and not providing it to patients outside the clinical trials, it is important to understand the following:

Rule One: The company or individual who owns the drug has to agree to give it to you. No one can make them provide the unapproved drug outside the clinical trial — not you, your doctor, the FDA, your Senator or even the President. This is a very important point that is widely misunderstood.

Rule Two: Drug companies vary tremendously in their policies regarding compassionate use. Some offer compassionate use for one drug but not for another drug. Some are much more open to the idea than others and some are more organized than others about handling such requests. Some will just decide not to return your calls.

Talk to the right person about Single Patient Access

If a program or policy about compassionate use doesn't exist, you'll need to push. The person you'll want to reach is the Medical Director for Oncology Clinical Development for the specific drug you're interested in. Getting to him or her on the phone will probably take persistence and creativity. Getting what you want once you do so may be even harder.

If you run into a dead end, your doctor may have better luck reaching the right person. Do the legwork before asking your doctor to make the call. For example, ask your doctor for a time it would be convenient for him to call the company and schedule that time with the company contact. If possible, go to your doctor's office the day and time he places the call to make sure it happens and learn the result.

You may also want to contact the staff in FDA's Office of Special Health Issues (301-827-4460) who can assist you in determining how to approach a drug company. However, remember that the FDA can not call a drug company on your behalf because the company could construe that as pressure or coercion. The FDA "allows" but does not encourage compassionate use. Drug companies must consider their own circumstances and reach their own conclusion about the advisability of sponsoring a compassionate use program or allowing a single patient to have access to their unapproved drug.

Meeting FDA requirements for Single Patient Access

Remember, you will either be given the new drug as an individual - Single Patient Access, or as part of a group - Expanded Access Program (EAP). In an EAP, the clinical researchers or their staff at the site running the EAP will walk you through the required process.

In every case, your use of an unapproved drug outside a clinical trial must be approved by the FDA.

Once the drug company has agreed to provide the drug to you through your doctor, the FDA's Office of Special Health Issues - Cancer Liaison Program staff (301-827-4460) can help determine next steps.

The FDA's website has a page that explains exactly how your doctor can apply for what's called a "single patient exception."

What happens if demand for the drug is greater than the supply? How does a company decide which patients will receive the drug?

The fact is there are as many answers to this question as there are drug companies.

A drug company might allow the physician researchers (also known as investigators) at each clinical trial site to select a few promising patients to be given the new drug outside of the clinical trial. This is called a site-specific protocol exemption.

Occasionally, companies sponsor a lottery. In other words, the patient applicants' name is placed into a computer and the computer randomly selects the names of the patients who will receive the drug for each day, week or month — depending on the selection frequency schedule. The lottery selection is preferred by some drug companies and patient advocates because the selection is unbiased and each patient applicant in the lottery pool has an equal chance of being selected.

In some cases, drug companies, largely because of inexperience, allow Single Patient Access of their drug without thinking through the advisability of managing patient requests one at a time. As a result, the company may become overwhelmed by the demand and then begin to deny Single Patient Access requests.

And finally, a drug company's process for managing Single Patient Access requests may seem almost random: a company will say yes to one person, while others will be denied without a clear basis for why either answer was given to the patient.

Summary

The best way to get access to an unapproved drug is through a clinical trial. Expanded access programs, if offered by the drug company, offer another option. If neither of those two options exists, access through single-patient compassionate use may be possible.

Working out single-patient compassionate use of an unapproved drug will almost certainly be confusing, time-consuming and frustrating. There is no single policy or process adhered to by the FDA and drug companies. There is no single list of drugs currently available through compassionate use. There is no way to force the drug company or FDA to permit treatment through compassionate use.